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ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD)

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ClinicalTrials.gov Identifier: NCT04363684
Recruitment Status : Recruiting
First Posted : April 27, 2020
Last Update Posted : June 8, 2021
Sponsor:
Collaborators:
University of California, San Francisco
National Institute on Aging (NIA)
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
Bradley Boeve, Mayo Clinic

Tracking Information
First Submitted Date April 2, 2020
First Posted Date April 27, 2020
Last Update Posted Date June 8, 2021
Actual Study Start Date March 1, 2020
Estimated Primary Completion Date July 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 24, 2020)
  • Change in Brain Volumes [ Time Frame: Baseline, 1 Year, 2 Year, 3 Year, 4 Year, 5 Year ]
    Compare rates of change in whole brain and regional volumes between asymptomatic f-FTLD and symptomatic f- and s-FTLD, measured using MRI.
  • Change in NIH Examiner Executive Composite Score [ Time Frame: Baseline, 1 Year, 2 Year, 3 Year, 4 Year, 5 Year ]
    Evaluate change in NIH Examiner Executive Composite Score in asymptomatic f-FTLD.
  • Change in Multidomain Impairment Rating (MIR) Scale [ Time Frame: Baseline, 1 Year, 2 Year, 3 Year, 4 Year, 5 Year ]
    Annual change in MIR score (total score 0-3), which is a new global scale for FTLD that incorporates behavioral, cognitive, and motor dysfunction in the rating.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: April 24, 2020)
Plasma Neurofilament Light Chain Analysis [ Time Frame: 5 years ]
Annual blood samples will be collected to detect changes in plasma neurofilament light chain concentrations
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD)
Official Title ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD)
Brief Summary ARTFL LEFFTDS Longitudinal Frontotemporal Lobar Degeneration (ALLFTD) represents the formalized integration of ARTFL (U54 NS092089; funded through 2019) and LEFFTDS (U01 AG045390; funded through 2019) as a single North American research consortium to study FTLD for 2019 and beyond.
Detailed Description The ARTFL LEFFTDS Longitudinal Frontotemporal Dementia (ALLFTD) study aims to evaluate sporadic (s-) and familial (f-) frontotemporal lobar degeneration (FTLD) patients and asymptomatic family members of f-FTLD patients, characterizing the cohorts longitudinally and informing clinical trial design. The study has two arms: a "longitudinal arm" involving a comprehensive assessment of clinical, functional, imaging, and biofluid data collection annually, and a "biofluid-focused arm" involving limited clinical data to accompany biospecimen collection. For more information: https://www.allftd.org/
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
DNA, RNA, plasma, serum, PBMC, CSF (CSF is optional)
Sampling Method Non-Probability Sample
Study Population Participants will have a referring diagnosis of an FTLD clinical syndrome or will be a member of a family with a strong family history of an FTLD syndrome.
Condition
  • Frontotemporal Lobar Degeneration (FTLD)
  • Progressive Supranuclear Palsy (PSP)
  • Corticobasal Degeneration (CBD)
  • Behavioral Variant Frontotemporal Dementia (bvFTD)
  • Semantic Variant Primary Progressive Aphasia (svPPA)
  • Nonfluent Variant Primary Progressive Aphasia (nfvPPA)
  • FTD With Amyotrophic Lateral Sclerosis (FTD/ALS)
  • Amyotrophic Lateral Sclerosis
  • Oligosymptomatic PSP (oPSP)
  • C9orf72
  • GRN Related Frontotemporal Dementia
  • MAPT Gene Mutation
  • TBK1 Gene Mutation
  • Oligosymptomatic Progressive Supranuclear Palsy
Intervention Not Provided
Study Groups/Cohorts
  • Longitudinal Arm
    Annual clinic visits throughout the length of the study.
  • Biofluid-Focused Arm
    Single clinic visit.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: April 24, 2020)
2100
Original Estimated Enrollment Same as current
Estimated Study Completion Date July 2024
Estimated Primary Completion Date July 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria

Longitudinal Arm Inclusion Criteria

Familial FTLD (f-FTLD) participants (either is acceptable):

  • members of families in whom at least one member has a known disease-associated mutation in one of the major genes that cause f-FTLD: MAPT, GRN, C9orf72 (or other rare genes)
  • an autosomal dominant family history of a FTLD syndrome (without a known gene) verified by medical record review or well-documented family history including family members with a medical history consistent with FTLD or a related disorder.

Sporadic FTLD (s-FTLD) participants:

Sporadic participants should be symptomatic with no known family history nor a genetic mutation indicating f-FTLD. All sporadic participants must have an FTLD syndrome as a referring diagnosis; those determined by ALLFTD clinicians to have non-FTLD diagnoses will be excluded from longitudinal visits, but their baseline visit will be included in comparative datasets. For inclusion in the longitudinal follow-up, participants should meet research criteria for one of the following FTLD syndromes:

  • Progressive Supranuclear Palsy (PSP)
  • Semantic variant Primary Progressive Aphasia (svPPA)
  • Nonfluent variant Primary Progressive Aphasia (nfvPPA)
  • Corticobasal Degeneration (CBD)/Corticobasal Syndrome (CBS)
  • Behavioral variant Frontotemporal dementia (bvFTD)
  • Frontotemporal Dementia with Amyotrophic Lateral Sclerosis (FTD/ALS)

Biofluid-Focused Arm Inclusion Criteria

Participants enrolled in the biofluid arm may be either f-FTLD or s-FTLD. All general inclusion criteria apply. Participants should meet research criteria (as specified above) for any FTLD syndrome or meet familial FTLD inclusion criteria. Because the biofluid arm participants do not undergo the same detailed clinical and functional assessments required for the longitudinal arm, participants may be included regardless of primary language, as long as an appropriately translated consent is available.

Exclusion Criteria:

  • Known presence of a structural brain lesion (e.g. tumor, cortical infarct) that could reasonably explain symptoms in a symptomatic participant.
  • Known presence of an Alzheimer's disease causing mutation in PSEN1, PSEN2 or APP; or biomarker evidence for Alzheimer's disease as a cause of the clinical syndrome.
  • A previous history of Korsakoff encephalopathy, severe alcohol dependence (within 5 years of onset of dementia), frequent alcohol or other substance intoxication, or other neurological disorder.
  • Evidence through history or laboratory testing of uncorrected B12 deficiency (B12 < 95% of local laboratory's normal value), unregulated hypothyroidism (TSH >150% of normal), HIV positive, renal failure (creatinine > 2), liver failure (ALT or AST > two times normal), respiratory failure that requires supplemental oxygen, large confluent white matter lesions, significant systemic medical illnesses such as deteriorating cardiovascular disease.
  • Current medication likely to affect CNS functions in the opinion of the site PI.
  • In the site investigator's opinion, the participant cannot complete sufficient key study procedures. The participant may be enrolled into the biofluid-focused arm if they can tolerate a blood draw and short clinical exam, but must be able to complete at least 75% of study procedures for enrollment into the longitudinal arm.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers Yes
Contacts
Contact: Leah K Forsberg, PhD 507-293-9577 forsberg.leah@mayo.edu
Contact: Hilary Heuer, PhD 415-476-6743 hilary.heuer@ucsf.edu
Listed Location Countries Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04363684
Other Study ID Numbers 19-004543
U19AG063911 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: Yes
Plan Description: De-identified subject level data will be shared upon approved data request.
Time Frame: De-identified data will be available for at least the duration of the study.
Access Criteria:

Interested researchers must complete a data request through the ALLFTD website. All data requests will be reviewed by a committee for evaluation of scientific merit and feasibility. Please consult the website for additional information regarding this process (https://www.allftd.org/policies).

Approved requests will be delivered in a de-identified manner.

URL: https://www.allftd.org/data
Responsible Party Bradley Boeve, Mayo Clinic
Study Sponsor Mayo Clinic
Collaborators
  • University of California, San Francisco
  • National Institute on Aging (NIA)
  • National Institute of Neurological Disorders and Stroke (NINDS)
Investigators
Principal Investigator: Bradley Boeve, MD Mayo Clinic
Principal Investigator: Adam Boxer, MD, PhD University of California, San Francisco
Principal Investigator: Howie Rosen, MD University of California, San Francisco
PRS Account Mayo Clinic
Verification Date June 2021